AX200 (recombinant human G-CSF; Filgrastim) displays neuroregenerative and neuroprotective properties in various preclinical models. Based on a comprehensive preclinical data set which showed unique therapeutic benefits of G-CSF in different experimental stroke models, the AXIS 1 clinical trial was conducted. AXIS 1 stands for AX200 treatment in acute ischemic stroke. In this multi-centre, randomized, double-blind, placebo-controlled dose escalation trial 44 patients were enrolled and four different doses of recombinant human G-CSF (Filgrastim) were tested. The stroke patients received intravenous Filgrastim (or placebo) in the first 12 hours after stroke for three days. AXIS 1 met its clinical endpoints demonstrating safety and tolerability of high doses of Filgrastim in acute stroke patients. As expected, a dose related increase in white blood cell count was seen, providing a good rationale to select the dose for the AXIS 2 efficacy study.
Multivariate analyses showed hints for efficacy in particular in patients with larger infarcts. Scales used in this analysis included the National Institutes of Health Stroke Scale (NIHSS), the Barthel Index (BI) and the modified Rankin Scale (mRS).
Further information on the clinical trial can be obtained at http://clinicaltrials.gov/ct/show/NCT00132470.